Sarepta Therapeutics, Inc.: Muscular Dystrophies trials

The registry lists 19 Muscular Dystrophies studies with Sarepta Therapeutics, Inc. as lead sponsor. 0 are open to participants now and 16 have results posted.

Phase 1 8Phase 2 13Phase 3 3No phase 1(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT05096221Phase 3Results posted
• A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)TerminatedNCT03985878Phase 2Results posted
• A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)CompletedNCT03769116Phase 1/2Results posted
• An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)TerminatedNCT03675126Phase 1/2Results posted
• An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyTerminatedNCT03532542Phase 3Results posted
• A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT03375164Phase 1/2Results posted
• Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 SkippingCompletedNCT03218995Phase 2Results posted
• Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) PatientsCompletedNCT02530905Phase 1Results posted
• Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyCompletedNCT02420379Phase 2Results posted
• Phase I/II Study of SRP-4053 in DMD PatientsCompletedNCT02310906Phase 1/2Results posted
• Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyCompletedNCT02286947Phase 2Results posted
• Study of Eteplirsen in DMD PatientsCompletedNCT02255552Phase 3Results posted
• A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)CompletedNCT01976091Phase 1/2Results posted
• Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyCompletedNCT01540409Phase 2Results posted
• Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsCompletedNCT01396239Phase 2Results posted
• Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsCompletedNCT00844597Phase 1/2Results posted
• A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical PracticeActive, not recruitingNCT04475926
• Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentTerminatedNCT04004065Phase 2
• A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)CompletedNCT03375255Phase 1